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Research Highlight | Jan 3, 2017

Personalizing treatment for patients with the most common malignant childhood brain tumour

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A TFRI-funded research team studying the most common type of brain tumour in children recently demonstrated the benefits of personalizing treatment for children with medulloblastoma.

With a mortality rate of 30 to 40 per cent among children who are diagnosed with the disease, those who do survive often experience negative side effects from surgery, radiation, and chemotherapy.

The research, led by the lab of Dr. Donald Mabbott and his graduate student Iska Moxon-Emre at Toronto’s SickKids Hospital, was published in the Journal of Clinical Oncology http://ascopubs.org/doi/full/10.1200/JCO.2016.66.9077 (August 2016). The paper suggests that children with less aggressive subtypes of medulloblastoma had spared intelligence -- and their survival was not compromised -- when they received less radiation.

For the study, data from 121 children who were treated between 1991 and 2013 at the Hospital for Sick Children (Toronto,Ontario), Children’s National Health System (Washington, DC), or the Lucile Packard Children’s Hospital (Palo Alto, CA) was compared.

They found that children in two of four subgroups -- WNT and Group 4 -- benefitted from less aggressive treatment, and there was no increase in mortality. Further, the evidence suggests that subgrouping patients based on their genetics can help identify those with lower-risk disease who may be candidates for less aggressive therapy to spare their cognitive function, keeping both survival rates and functionality high.

Interestingly, patients with the sonic hedgehog (SHH) subtype had the lowest incidence of mutism and motor deficits, common post-surgical complications. This finding indicates that the subgroups differ in their functional outcomes, and further highlights the value of using subgroup information to help guide treatment for patients with medulloblastoma.

This was the first study of its kind to report on intellectual outcome in molecular subgroups of medulloblastoma, and the results promise to benefit future patients.

StudyIntellectual outcome in molecular subgroups of medulloblastoma

AuthorsIska Moxon-Emre, Michael D. Taylor, Eric Bouffet, Kristina Hardy, Cynthia J. Campen, David Malkin, Cynthia Hawkins, Normand Laperriere, Vijay Ramaswamy, Ute Bartels, Nadia Scantlebury, Laura Janzen, Nicole Law, Karin S. Walsh, and Donald J. Mabbott 

FundingSupported by Canadian Institutes of Health Research, Brain Canada, Pediatric Oncology Group of Ontario, and the MAGIC (Medulloblastoma Advanced Genomics International Consortium) project. The MAGIC project acknowledges funding from Genome Canada, Genome British Columbia, Terry Fox Research Institute, Ontario Institute for Cancer Research, Pediatric Oncology Group Ontario, the family of Kathleen Lorette, Clark H. Smith Brain Tumour Centre, Montreal Children’s Hospital Foundation, Hospital for Sick Children, Sonia and Arthur Labatt Brain Tumour Research Centre, Chief of Research Fund, Cancer Genetics Program, Garron Family Cancer Centre, and B.R.A.I.N. Child (Brain Tumour Research Assistance and Information Network).

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